World’s most expensive drug: a drug against muscle disease admitted to the

The Swiss pharmaceutical company Novartis has received US FDA approval for a Gene therapy for the treatment of a rare muscle disease. The dose of the most expensive drug in the world is about to cost two million dollars.

A single dose costs more than two million dollars

The Swiss pharmaceutical group Novartis has received on Friday by the US-American health authority FDA (Food and Drug Administration) approval for a Gene therapy for the treatment of a rare muscle disease. The price of the drug immediately sparked a controversy over the rising cost of medicines and payment. Because: A single dose of the drug should cost 2,125 million dollars.

For the treatment of a genetically-muscle-related disease

How different types of media such as the news channel “CNN” reports, is marketed gene therapy called Zolgensma of AveXis, whose parent company is Novartis.

The FDA approved the use of the Drug in children under two years of age for the treatment of the genetic disease spinal muscular atrophy (SMA).

The genetic disease leads to progressive atrophy of muscles, and to an early death or life-long disability.

“In Germany there are about 5,000 Affected in all ages, and approximately 2 million people are Carriers for SMA,” explains the German muscle Foundation on your website.

“Worldwide, the Numbers, therefore, are at more than 375,000 people Affected and 150 million people are Carriers”, and more.

Controversy surrounding the rising costs of medicines

The resources of Novartis will help to be Concerned.

“The potential of gene therapy products that change the lives of patients who may be Suffering from a deadly, or worse in front of the death, gives hope for the future,” said FDA Commissioner Dr. Ned Sharpless in a message.

The price of the drug and caused an immediate controversy surrounding the rising costs of medicines and their payment.

“It is a Symbol of our broken System that forces us to pay the price, to the pharmaceutical companies for life-saving new medicines, demand,” said David Mitchell, founder of the advocacy “Patients For Affordable Drugs,” according to “CNN”.

The managers of Novartis, said that the price had reached the target cost efficiency. The newly approved treatment cost is about half what it could cost another treatment for the disease, Spinraza, for over ten years.

The company expects this year, with the approval of the drug in Europe and Japan.

Currently applied therapy

The spinal muscular atrophy (SMA) is currently treated with Spinraza of the US biotechnology company Biogen.

“The drug needs intrathecal be administered, i.e., by a lumbar puncture (CSF collection in the same amount as the drug is supplied). This needs to be repeated regularly,“ explains the German society for Muscle disorders (DGM) on your website.

“The treatment regime provides for a application at a distance from the first 14 days, then 30 and 60 days and then every four months”, – stated there.

According to the news Agency Reuters, the treatment in the first year of Treatment of 750,000 US dollars, and in consequence of cost of 375,000 dollars per year.

The data suggest that Novartis had provided for now in the US approved the Drug last in a price range of 1.5 to five million dollars in the room. (ad)